The US Food and Drug Administration (FDA) has approved Rocket Pharmaceuticals' Kresladi (marnetegragene autotemcel), the first gene therapy for the treatment of severe Leukocyte Adhesion Deficiency Type I (LAD-I).
Kresladi is indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant.
Severe LAD-I is a rare, inherited immune deficiency caused by mutations in the ITGB2 gene, which prevent white blood cells from effectively fighting infections.
Kresladi consists of the patient’s own hematopoietic (blood) stem cells (HSCs), which are genetically modified to introduce functional copies of the ITGB2 gene. Following conditioning, a single dose of Kresladi is infused intravenously to address the underlying cause of severe LAD-I by restoring CD18 and CD11a cell surface expression in white blood cells, including neutrophils.
